Company Product Description Indication Status
Phase I
Bioeclipse Therapeutics Inc., of Mountain View, Calif. CRX-100 Combines cytokine-induced killer cells with an oncolytic virus Refractory solid tumors Started patient enrollment in dose-escalation study
Genkyotex SA, of Archamps, France Setanaxib NOX1 and NOX4 inhibitor Primary biliary cholangitis Data showed drug was well-tolerated in healthy adults at doses up to 1,600 mg/day and displayed dose-proportional exposure; no safety signal or dose-limiting toxicity identified across doses tested
Grifols SA, of Barcelona, Spain COVID-19 candidate Containing immunoglobulin Gamunex-C and anti-SARS-CoV-2 polyclonal antibodies from plasma donors who recovered from disease COVID-19 Set to start study in Spain
Lixte Biotechnology Holdings Inc., of East Setauket, N.Y. LB-100 Protein phosphatase inhibitor Small-cell lung cancer Entered agreement with City of Hope for phase Ib trial in combination with standard regimen for untreated, extensive-stage disease
Sernova Corp., of London, Ontario Cell Pouch Cell therapy Type 1 diabetes Additional preliminary data from 5 of 7 patients currently enrolled showed 2 who had received second islet transplant had sustained blood levels of C-peptide, reduction of HbA1c and improvement in overall continuous glucose monitoring
Sinomab Bioscience Ltd., of Hong Kong SN-1011 Third-generation covalent reversible BTK inhibitor Immunological diseases First healthy subject dosed
Vallon Pharmaceuticals Inc., of Philadelphia Adair  Oral formulation of immediate-release dextroamphetamine formulated to be abuse-deterrent Attention deficit hyperactivity disorder and narcolepsy Pharmacokinetics and pharmacodynamic data from 2 studies; 1 showed geometric LS means and 90% confidence intervals of Cmax, AUC0-t and AUC0-∞ all met predefined acceptance criteria; second study demonstrated manipulated intranasal Adair had significant reduction in drug liking (p<0.0001) compared with intranasal d-AMPH
Phase II
Aclaris Therapeutics Inc., of Wayne, Pa. ATI-450 Oral MK2 inhibitor Moderate to severe rheumatoid arthritis Top-line data from phase IIa study in 19 subjects receiving drug or placebo in combination with methotrexate for 12 weeks showed mean change from baseline in DAS28-CRP score was 2 reduction in the treatment arm compared to a 0.35 increase in placebo arm; proportion of subjects with a DAS28-CRP score of ≤ 3.2 (low disease activity or remission) was 40% and 0% in the treatment and placebo arms, respectively, and proportion of subjects with a DAS28-CRP score of < 2.6 (remission) was 20% and 0% in the treatment and placebo arms, respectively
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia NP-120 (ifenprodil) Noncompetitive NMDA antagonist COVID-19 Data and safety monitoring board completed latest review of phase IIb part of phase IIb/III trial and provided approval for firm to start phase III portion
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Troriluzole Neuroprotectant Mild to moderate Alzheimer’s disease Analysis of top-line data from phase II/III study showed drug did not statistically differentiate from placebo at 48 weeks on prespecified co-primary endpoints on the Alzheimer's Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes; drug also did not differentiate from placebo on key secondary measure of hippocampal volume assessed by MRI in the overall population; subgroup analysis of mild AD patient showed nonsignificant numerical difference of a potential benefit at week 48 on both the ADAS-cog and hippocampal volumetric MRI
Biophytis SA, of Paris Sarconeos (BIO-101) Proto-oncogene Mas agonist COVID-19 Data monitoring committee recommended recruitment start for part 2 of phase II/III Cova study assessing Sarconeos in acute respiratory failure associated with COVID-19; results are expected in second quarter of 2021
Caladrius Biosciences Inc., of Basking Ridge, N.J. CLBS-16 Autologous CD34+ cells Coronary microvascular dysfunction Treated first patient in phase IIb Freedom trial
Debiopharm SA, of Lausanne, Switzerland Alisporivir (Debio-025) Cyclophilin inhibitor COVID-19 Dosed first patient in investigator-initiated study in early stage, hospitalized patients not requiring medical ventilation or exhibiting signs of acute respiratory distress syndrome; primary objective is to evaluate reduction in COVID-19 viral load 
Evgen Pharma plc, of Cheshire, U.K. SFX-01 Up-regulates Nrf2 pathway Acute respiratory infections As of Jan. 18, 2021, 56 patients recruited to Star phase II/III trial testing drug’s ability to prevent onset of acute respiratory distress syndrome in patients with community-acquired pneumonia from any infective agent and who have been or are being tested for suspected COVID-19
Incarda Therapeutics Inc., of San Francisco Inrhythm (flecainide for inhalation) E3 ubiquitin protein ligase COP1 inhibitor Recent-onset paroxysmal atrial fibrillation Dosed first U.S. patient in Instant study
Servier SA, of Paris Lonsurf (trifluridine/tipiracil) Thymidine-based nucleoside analogue and thymidine phosphorylase inhibitor Unresectable metastatic colorectal cancer Updated results from Tasc01 study testing combination with bevacizumab and capecitabine in first-line patients who are non-eligible for intensive therapy showed median overall survival was 22.3 months vs. 17.6 months with bevacizumab/capecitabine, which translated into an improved median OS of 4.6 months
Sirnaomics Inc., of Gaithersburg, Md. STP-705 siRNA designed to knock down TGF-β1 and COX-2 gene expression Cutaneous basal cell carcinoma Dose administration for first patient in phase IIa study; objective is to determine safe and effective recommended dose, as well as analysis of biomarkers common to BCC formation pathway, including TGF-β1 and COX-2
Phase III
Agios Pharmaceuticals Inc., of Cambridge, Mass. Tibsovo (ivosidenib tablets) IDH1 gene inhibitor IDH1-mutated cholangiocarcinoma Full analysis of final data from Claridhy trial in patients with previously treated disease showed an improvement in secondary endpoint of overall survival favoring Tibsovo arm vs. placebo, though statistical significance was not reached; median OS for Tibsovo was 10.3 months vs. 7.5 months for placebo (1-sided p=0.093)
Bausch + Lomb, of Laval, Quebec, and Novaliq GmbH, of Heidelberg, Germany NOV-03 Perfluorohexyloctane ophthalmic solution Dry eye disease associated with Meibomian glad dysfunction First of 2 studies has been completely enrolled, with total of 599 participants
Brickell Biotech Inc., of Boulder, Colo., and Kaken Pharmaceutical Co. Ltd., of Tokyo BBI-4000  Sofpironium bromide gel Primary axillary hyperhidrosis Pivotal data from Japanese trial published in the Journal of Dermatology showed proportion of patients who achieved primary endpoint was 53.9% vs. 36.4% in vehicle group (p=0.003)
Phathom Pharmaceuticals Inc., of Florham Park, N.J. Vonoprazan Oral small-molecule potassium-competitive acid blocker H. pylori infection Completed patient enrollment in Phalcon-HP pivotal study testing combination with amoxicillin and combination with amoxicillin and clarithromycin; top-line data expected in second quarter of 2021
Verrica Pharmaceuticals Inc., of West Chester, Pa. VP-102 Drug-device combination containing GMP-controlled formulation of cantharidin Molluscum contagiosum Data from post-hoc pooled analysis of pivotal Camp trials showed treated participants with lesions in the upper extremities, head/neck, back/buttocks and chest/abdomen at baseline showed statistically significantly higher rates of complete clearance in those regions vs. vehicle beginning after the first treatment (visit 2; day 21) through the end-of-treatment visit; VP-102-treated participants with lesions in the lower extremities at baseline showed statistically significantly higher complete clearance rates compared to vehicle-treated participants beginning after 2 treatments (visit 3; day 42) through the EOS visit (day 84)


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